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TerminatedNCT00617890Updated Aug 23, 2018Results posted

A Study to Determine the Activity of Robatumumab (SCH 717454) in Participants With Relapsed Osteosarcoma or Ewing's Sarcoma (MK-7454-002/P04720)

A Phase 2 interventional study of robatumumab in Osteosarcoma, Sarcoma, Ewing's and Peripheral Neuroectodermal Tumor, sponsored by Merck Sharp & Dohme LLC. Terminated. Open to participants aged 4 Years and older. Per ClinicalTrials.gov, last updated 2018-08-23.

Sponsored by Merck Sharp & Dohme LLC · Phase 2, Interventional, and Treatment

Why this study was terminated
The study was prematurely terminated for strategic reasons, not for a safety concern.
Phase
Phase 2
Study type
Interventional
Enrollment
219
Allocation
Randomized
Ages
4 Years and older
Sex
All
01

Study summary

Participants with relapsed osteosarcoma that can be treated with surgery will be randomized to robatumumab administered intravenously (IV) at one of two dose levels. These participants will first receive robatumumab, have surgery performed, and continue to receive treatment every two weeks until a year of dosing, or until disease progression.

Participants with unresectable osteosarcoma or Ewing Sarcoma will receive robatumumab IV once every two weeks until disease progression. Participants who achieve a complete response (CR) or partial response (PR) after tumor evaluations may undergo surgical resection. After surgery, participants are eligible to receive 10 mg/kg robatumumab until disease recurrence/progression or one year of total dosing, whichever occurs first.

Read the detailed description

Participants with resectable osteosarcoma will be randomized to one of two dose levels of robatumumab to be given intravenously. These participants will first receive robatumumab according to randomized treatment, and have surgery performed 10 to 14 days after initial dosing. Participants will be allowed to recover from surgery four to six weeks prior to additional robatumumab administration at their randomized dose level. robatumumab will then be administered on the same calendar day once every two weeks. Participants will continue to receive robatumumab until disease recurrence, or until completing a year of dosing at the same dose level assigned, whichever occurs first.

Participants with unresectable osteosarcoma or Ewing Sarcoma will be assigned treatment to robatumumab IV administered once every two weeks and will continue to receive robatumumab until disease progression. Participants who achieve a CR or PR after tumor evaluations may undergo surgical resection. After surgery, participants are eligible to receive 10 mg/kg robatumumab until disease recurrence/progression or one year of total dosing, whichever occurs first.

02

Conditions studied

03

Who can participate

Ages eligible
4 Years and older
Sexes eligible
All
Accepts healthy volunteers
No

Inclusion criteria

  • A participant must be 11 years of age or older and may be of any race, and gender; participants between 4 and 10 years of age, inclusive, may be considered on a site-by-site basis.
  • A participant must have a diagnosis of histologically confirmed osteosarcoma or Ewing sarcoma;
  • A participant with either:

    • relapsed and resectable osteosarcoma
    • relapsed and unresectable osteosarcoma that is refractory to standard therapy, ie. has relapsed after prior systemic treatment with active chemotherapy agents
    • Ewing sarcoma that is refractory to standard systemic therapies
  • A participant >16 years of age must have an Eastern Cooperative Oncology Group (ECOG) performance status of \<=2; a participant \<=16 years of age must have a Karnofsky performance status between 50% and 100% or a Lansky play scale between 50% and 100%
  • A participant must have adequate organ function.

Exclusion criteria

Exclusion Criteria:

  • A participant with a history of another malignancy (with the exception of non-melanoma skin cancer or carcinoma in situ of the cervix treated with curative intent at least 2 years prior to start of treatment, or other adequately treated malignancy for which the subject has been disease free for >=5 years)
  • A participant who has known treated or untreated leptomeningeal metastasis, or a metastatic central nervous system lesion
  • A participant with a history of uncontrolled diabetes mellitus
  • A participant with a recent myocardial infarction (within the past year); or a participant who at the time of Screening presents with unstable or uncontrolled angina, New York Heart Association (NYHA) Class III or IV congestive heart failure, uncontrolled hypertension, clinically significant cardiac dysrhythmia or clinically significant electrocardiogram (ECG) abnormality
  • A participant with an active infection
  • A participant with clinically significant hepatitis at Screening, or a participant who is hepatitis C antibody positive, hepatitis B surface antigen positive, or human immunodeficiency virus (HIV) seropositive
  • A participant who has been treated with an anti-insulin-like growth factor receptor 1 (anti-IGF-1R)- targeted drug or antibody
  • A participant with known hypersensitivity to other antibodies, or any accompanying excipients associated with these medications.
04

Study design

Phase
Phase 2
Primary purpose
Treatment
Allocation
Randomized
Intervention model
Parallel assignment
Masking
Double (Participant, Investigator)
Enrollment
219 participants (actual)

Study arms

  • Experimental
    Group 1: 0.3 mg/kg

    Participants received robatumumab 0.3 mg/kg intravenously (IV) as a single dose on Day 1, followed by surgery on Day 10 to 14, and four weeks later, resumption of robatumumab 0.3 mg/kg on the same calendar day (± 3 days) once every 2 weeks until disease recurrence or up to 1 year of dosing. This group comprised participants with resectable osteosarcoma that relapsed within 6 months of prior definitive treatment (eg surgical metastasectomy) and having at least one prior chemotherapy regimen containing a platinum agent and doxorubicin.

    Biological: robatumumab

  • Experimental
    Group 1: 10 mg/kg

    Participants who received robatumumab 10 mg/kg IV as a single dose on Day 1, followed by surgery on Day 10 to 14, and four weeks later, resumption of robatumumab 10 mg/kg on the same calendar day (± 3 days) once every 2 weeks until disease recurrence or up to 1 year of dosing. This group comprised participants with resectable osteosarcoma that relapsed within 6 months of prior definitive treatment (eg surgical metastasectomy) and having at least one prior chemotherapy regimen containing a platinum agent and doxorubicin.

    Biological: robatumumab

  • Experimental
    Group 2: 10 mg/kg

    Participants received robatumumab 10 mg/kg IV biweekly until disease recurrence or up to 1 year of dosing. This group comprised participants with relapsed and unresectable osteosarcoma refractory to prior chemotherapy with a platinum- and doxorubicin-containing regimen.

    Biological: robatumumab

  • Experimental
    Group 3: 10 mg/kg

    Participants received robatumumab 10 mg/kg IV biweekly until disease recurrence or up to 1 year of dosing. This group comprised participants with Ewing sarcoma refractory to prior treatment with at least 3 of the following agents: ifosfamide, etoposide, cyclophosphamide, doxorubicin, or vincristine.

    Biological: robatumumab

Interventions

  • Biologicalrobatumumab

    Robatumumab IV every two weeks until disease progression.

    Also known as: SCH 717454, SCH 717454 (19D12), MK-7454

05

What researchers measure

Primary outcomes

  1. Number of Participants Achieving a Complete Response or Partial Response (Group 3 Only)

    This is a measure of the number of participants with a complete response (CR) or partial response (PR) to therapy, confirmed by central review. Response was based on Response Evaluation Criteria in Solid Tumors (RECIST) and World Health Organization (WHO) criteria.

    Time frame: Up to 1 year following the start of study therapy

  2. Number of Participants With >= 25% Change in Tumor Proliferation After Exposure to Robatumumab (Group 1 Only)

    Tumor proliferation was measured using Ki-67 levels. Ki-67 is nuclear protein associated with cellular proliferation.

    Time frame: Approximately 14 days

  3. Number of Participants Achieving a Complete Response, a Partial Response, or Stable Disease (Group 2 Only)

    Responses to treatment (complete response, partial response, or stable disease) confirmed by central review for Participants in Group 2. Response was based on Response Evaluation Criteria in Solid Tumors (RECIST) and World Health Organization (WHO) criteria.

    Time frame: Up to 1 year following the start of study therapy

Secondary outcomes

  1. Overall Survival

    This is a measure of the number of participants known to be alive at the time of data analysis for this study.

    Time frame: From start of treatment until death or data analysis cut off (Up to 3.4 years)

  2. Time Until Tumor Relapse (Group 1 Only)

    This is a measure of the time from the start of the study to documented relapse of disease.

    Time frame: From start of treatment until relapse or data analysis cut off (Up to 3.4 years)

  3. Area Under the Concentration-time Curve (AUC) of Serum Levels of Robatumumab (Group 1 Only)

    Time frame: End of infusion on Day 1, and then prior to surgery, before and after the 2nd, 3rd, and 8th doses (up to 20 weeks)

  4. Incidence of Anti-robatumumab Antibodies

    For biological agents, it is possible for the host (participant) to develop antibodies to the agent. This outcome measure was planned to find out the number of participants who developed the antibodies after treatment with robatumumab.

    Time frame: Up to 2 years

  5. Number of Participants Experiencing Treatment-Emergent Adverse Events

    An adverse event is any unfavorable and unintended change in the structure, function, or chemistry of the body whether or not considered related to the study treatment. Treatment-emergent adverse events are those that occur after participants have received study treatment, or existing adverse events that occurred during screening that increase in severity after study treatment. Adverse events in the Group 1: 0.3 mg/kg arm that occurred after switching to the 10 mg/kg dose are displayed under the originally assigned treatment.

    Time frame: Up to 2 years

  6. Time to Disease Progression (Groups 2 and 3 Only)

    This is a measure of the time from the start of the study to the time of documented disease progression.

    Time frame: From the start of treatment until disease progression or data analysis cut off (Up to 3.4 years)

  7. Overall Survival (Groups 2 and 3 Only)

    This is a measure of the time of survival from first dose to documentation of death

    Time frame: From start of treatment until death or data analysis cut off (Up to 3.4 years)

  8. Duration of Response (Groups 2 and 3 Only)

    This is a measure of the amount of time in which the tumor responded to therapy.

    Time frame: From time of documented response until disease progression or data analysis cut off (Up to 3.4 years)

06

Results

Posted Dec 11, 2015
Limitations and caveats
The study was stopped prematurely for administrative reasons; not all planned endpoints were analyzed.

Participant flow

Participant flow — Overall Study
MilestoneGroup 1: 0.3 mg/kgGroup 1: 10 mg/kgGroup 2: 10 mg/kgGroup 3: 10 mg/kg
Started353335116
Received treatment343334115
Completed4501
Not completed312835115
Withdrew: Treatment ongoing at data cut-off0005
Withdrew: Lack of efficacy23263097
Withdrew: Lost to follow-up0012
Withdrew: Withdrawal by subject4102
Withdrew: Protocol violation2100
Withdrew: Adverse event1038
Withdrew: Not treated1011

Outcome measures

PrimaryNumber of Participants Achieving a Complete Response or Partial Response (Group 3 Only)

This is a measure of the number of participants with a complete response (CR) or partial response (PR) to therapy, confirmed by central review. Response was based on Response Evaluation Criteria in Solid Tumors (RECIST) and World Health Organization (WHO) criteria.

Time frame:
Up to 1 year following the start of study therapy
Reported as:
Number · Participants
Number of Participants Achieving a Complete Response or Partial Response (Group 3 Only)
ParticipantsGroup 3: 10 mg/kg
Number of Participants Achieving a Complete Response or Partial Response (Group 3 Only)6
PrimaryNumber of Participants With >= 25% Change in Tumor Proliferation After Exposure to Robatumumab (Group 1 Only)

Tumor proliferation was measured using Ki-67 levels. Ki-67 is nuclear protein associated with cellular proliferation.

Time frame:
Approximately 14 days

No measurements were reported for this outcome.

PrimaryNumber of Participants Achieving a Complete Response, a Partial Response, or Stable Disease (Group 2 Only)

Responses to treatment (complete response, partial response, or stable disease) confirmed by central review for Participants in Group 2. Response was based on Response Evaluation Criteria in Solid Tumors (RECIST) and World Health Organization (WHO) criteria.

Time frame:
Up to 1 year following the start of study therapy
Reported as:
Number · Participants
Number of Participants Achieving a Complete Response, a Partial Response, or Stable Disease (Group 2 Only)
ParticipantsGroup 2: 10 mg/kg
Number of Participants Achieving a Complete Response, a Partial Response, or Stable Disease (Group 2 Only)6
SecondaryOverall Survival

This is a measure of the number of participants known to be alive at the time of data analysis for this study.

Time frame:
From start of treatment until death or data analysis cut off (Up to 3.4 years)
Reported as:
Number · Participants
Overall Survival
ParticipantsGroup 1: 0.3 mg/kgGroup 1: 10 mg/kgGroup 2: 10 mg/kgGroup 3: 10 mg/kg
Overall Survival1716728
SecondaryTime Until Tumor Relapse (Group 1 Only)

This is a measure of the time from the start of the study to documented relapse of disease.

Time frame:
From start of treatment until relapse or data analysis cut off (Up to 3.4 years)

No measurements were reported for this outcome.

SecondaryArea Under the Concentration-time Curve (AUC) of Serum Levels of Robatumumab (Group 1 Only)
Time frame:
End of infusion on Day 1, and then prior to surgery, before and after the 2nd, 3rd, and 8th doses (up to 20 weeks)

No measurements were reported for this outcome.

SecondaryIncidence of Anti-robatumumab Antibodies

For biological agents, it is possible for the host (participant) to develop antibodies to the agent. This outcome measure was planned to find out the number of participants who developed the antibodies after treatment with robatumumab.

Time frame:
Up to 2 years

No measurements were reported for this outcome.

SecondaryNumber of Participants Experiencing Treatment-Emergent Adverse Events

An adverse event is any unfavorable and unintended change in the structure, function, or chemistry of the body whether or not considered related to the study treatment. Treatment-emergent adverse events are those that occur after participants have received study treatment, or existing adverse events that occurred during screening that increase in severity after study treatment. Adverse events in the Group 1: 0.3 mg/kg arm that occurred after switching to the 10 mg/kg dose are displayed under the originally assigned treatment.

Time frame:
Up to 2 years
Reported as:
Number · Participants
Number of Participants Experiencing Treatment-Emergent Adverse Events
ParticipantsGroup 1: 0.3 mg/kgGroup 1: 10 mg/kgGroup 2: 10 mg/kgGroup 3: 10 mg/kg
Number of Participants Experiencing Treatment-Emergent Adverse Events313031112
SecondaryTime to Disease Progression (Groups 2 and 3 Only)

This is a measure of the time from the start of the study to the time of documented disease progression.

Time frame:
From the start of treatment until disease progression or data analysis cut off (Up to 3.4 years)

No measurements were reported for this outcome.

SecondaryOverall Survival (Groups 2 and 3 Only)

This is a measure of the time of survival from first dose to documentation of death

Time frame:
From start of treatment until death or data analysis cut off (Up to 3.4 years)
Reported as:
Median · Months
Overall Survival (Groups 2 and 3 Only)
MonthsGroup 2: 10 mg/kgGroup 3: 10 mg/kg
Overall Survival (Groups 2 and 3 Only)8.18 (2.96 to 10.58)6.93 (4.93 to 11.10)
SecondaryDuration of Response (Groups 2 and 3 Only)

This is a measure of the amount of time in which the tumor responded to therapy.

Time frame:
From time of documented response until disease progression or data analysis cut off (Up to 3.4 years)

No measurements were reported for this outcome.

Adverse events

Collected over Adverse events are reported from enrollment up to 5 weeks after the end of treatment (up to 2 years). Non-serious events are listed at a 5% frequency threshold.

Adverse event summary by group
GroupDeathsSeriousOther
Group 1: 0.3mg/kg—17/34 (50%)28/34 (82.4%)
Group 1: 10mg/kg—8/33 (24.2%)30/33 (90.9%)
Group 2: 10mg/kg—12/34 (35.3%)29/34 (85.3%)
Group 3: 10mg/kg—57/115 (49.6%)104/115 (90.4%)
Most frequent serious events
Showing 10 of 116
Most frequent serious events
EventGroup 1: 0.3mg/kgGroup 1: 10mg/kgGroup 2: 10mg/kgGroup 3: 10mg/kg
PNEUMOTHORAXRespiratory, thoracic and mediastinal disorders4/341/330/341/115
PAINGeneral disorders0/341/330/3411/115
RESPIRATORY FAILURERespiratory, thoracic and mediastinal disorders3/341/330/345/115
PYREXIAGeneral disorders1/341/330/347/115
FEBRILE NEUTROPENIABlood and lymphatic system disorders2/340/330/340/115
DYSPNOEARespiratory, thoracic and mediastinal disorders0/340/332/343/115
RESPIRATORY DISTRESSRespiratory, thoracic and mediastinal disorders0/340/330/346/115
THROMBOCYTOPENIABlood and lymphatic system disorders0/340/331/344/115
CHEST PAINGeneral disorders0/340/331/344/115
CONSTIPATIONGastrointestinal disorders0/341/330/342/115
Most frequent other events
Showing 10 of 83
Most frequent other events
EventGroup 1: 0.3mg/kgGroup 1: 10mg/kgGroup 2: 10mg/kgGroup 3: 10mg/kg
NAUSEAGastrointestinal disorders13/3412/335/3435/115
PROCEDURAL PAINInjury, poisoning and procedural complications13/346/331/342/115
PYREXIAGeneral disorders9/3411/336/3421/115
CONSTIPATIONGastrointestinal disorders9/349/333/3434/115
COUGHRespiratory, thoracic and mediastinal disorders10/347/336/3416/115
VOMITINGGastrointestinal disorders9/348/333/3425/115
HEADACHENervous system disorders9/347/337/3424/115
DIARRHOEAGastrointestinal disorders3/346/332/3429/115
DECREASED APPETITEMetabolism and nutrition disorders5/344/334/3427/115
FATIGUEGeneral disorders7/347/337/3423/115

Baseline characteristics

Three participants did not receive study treatment but are included in the baseline population.

Age, Continuous
Age, Continuous(Years)Group 1: 0.3 mg/kgGroup 1: 10 mg/kgGroup 2: 10 mg/kgGroup 3: 10 mg/kgTotal
Mean23.7 ± 15.520.1 ± 10.327.5 ± 15.324.6 ± 11.424.3 ± 12.8
Sex: Female, Male
Sex: Female, Male(Participants)Group 1: 0.3 mg/kgGroup 1: 10 mg/kgGroup 2: 10 mg/kgGroup 3: 10 mg/kgTotal
Female1413114381
Male21202473138
07

Study locations

No study locations are listed for this record.

08

References and documents

Publications

  • Anderson PM, Bielack SS, Gorlick RG, Skubitz K, Daw NC, Herzog CE, Monge OR, Lassaletta A, Boldrini E, Papai Z, Rubino J, Pathiraja K, Hille DA, Ayers M, Yao SL, Nebozhyn M, Lu B, Mauro D. A phase II study of clinical activity of SCH 717454 (robatumumab) in patients with relapsed osteosarcoma and Ewing sarcoma. Pediatr Blood Cancer. 2016 Oct;63(10):1761-70. doi: 10.1002/pbc.26087. Epub 2016 Jun 30. PubMed 27362300 ↗
  • Asmane I, Watkin E, Alberti L, Duc A, Marec-Berard P, Ray-Coquard I, Cassier P, Decouvelaere AV, Ranchere D, Kurtz JE, Bergerat JP, Blay JY. Insulin-like growth factor type 1 receptor (IGF-1R) exclusive nuclear staining: a predictive biomarker for IGF-1R monoclonal antibody (Ab) therapy in sarcomas. Eur J Cancer. 2012 Nov;48(16):3027-35. doi: 10.1016/j.ejca.2012.05.009. Epub 2012 Jun 7. PubMed 22682017 ↗

Individual participant data

Plan to share: Yes — https://www.merck.com/clinical-trials/pdf/ProcedureAccessClinicalTrialData.pdf

09

Registry details

Key details

Study ID
NCT00617890
Lead sponsor
Merck Sharp & Dohme LLC
Responsible party
Sponsor
First posted
Feb 18, 2008
Start date
Feb 1, 2008
Primary completion
Aug 31, 2011
Completion
Aug 31, 2013
Results posted
Dec 11, 2015
Last update
Aug 23, 2018

Study contacts

Medical Director
study director · Merck Sharp & Dohme LLC

Oversight

Data monitoring committee
No
View the source record on ClinicalTrials.gov ↗

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